Newly approved Nexviazyme (avalglucosidase alfa) is a next-generation, alternative enzyme replacement therapy for late-onset Pompe disease patients starting at age 1. In clinical trials that supported the Aug. 6 approval by the U.S. Food and Drug Administration (FDA), meaningful improvements in respiratory muscle strength and mobility were evident in patients given…

AT-GAA, an investigational therapy by Amicus Therapeutics for late-onset Pompe disease, will be available to select patients in the U.K. through an early access program before its potential regulatory approval. Eligible patients are adults who have received the enzyme replacement therapy (ERT) alglucosidase alfa — the…

Treatment with AT-GAA, an investigational therapy for late-onset Pompe disease, led to improvements in measures of physical and lung function in the Phase 3 clinical trial PROPEL, top-line data show. “Data from the PROPEL study demonstrate the potential to further improve motor and respiratory functions in patients with Pompe…

Lumizyme (alglucosidase alfa) is an enzyme replacement infusion therapy by Sanofi Genzyme for people with Pompe disease. In the European Union, it is available under the brand name Myozyme. Here are answers to some frequently asked questions about this treatment. How does Lumizyme work? Lumizyme works to replace…

Combining exercise training with a high-protein diet improves exercise tolerance, pulmonary function, and quality of life among late-onset Pompe disease (LOPD) patients taking Lumizyme (alglucosidase alfa) for years, a small study found. The study, “Exercise training alone or in combination with high-protein diet in patients…

Treatment with Myozyme (alglucosidase alfa, marketed as Lumizyme in the U.S.) improves walking ability and may stabilize respiratory function in adults with Pompe disease during the first three years of treatment, according to a recent study. However, factors such as aging may explain the loss of benefits observed at later stages. The…

Sanofi Genzyme’s next-generation enzyme replacement therapy (ERT), avalglucosidase alfa, safely halts disease progression in people with Pompe disease, according to interim data from two Phase 2 clinical trials. The trial’s results were the focus of oral and poster presentations at the 16th Annual WORLDSymposium, held…

The use of enzyme replacement therapy is safe and effective in Japanese people living with Pompe disease, a new study shows. Titled “Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance,” the study…

A woman with late-onset Pompe disease went through two successful pregnancies and births while continuing treatment with Lumizyme (alglucosidase alfa), a case study reports. The woman, however, chose not to breastfeed the infants. The study, “Two successfully completed pregnancies in adult onset Pompe disease,…

Treatment with Lumizyme (alglucosidase alfa) at double the approved dose may help prevent the rapid decline of respiratory and cardiovascular functions in infants with Pompe disease, a case report suggests. Since the U.S. Food and Drug Administration approved it in 2006, Lumizyme has change the course of Pompe…